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    EudraCT Number:2015-004333-27
    Sponsor's Protocol Code Number:SMTC11005
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-11-04
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2015-004333-27
    A.3Full title of the trial
    A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin
    Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with
    Duchenne Muscular Dystrophy (C11005)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to test how the study medication (SMT C1100) works and how safe it is when given to children with Duchenne Muscular
    A.3.2Name or abbreviated title of the trial where available
    PoC Study to Assess Activity and Safety of SMT C1100 in Boys with DMD
    A.4.1Sponsor's protocol code numberSMTC11005
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSummit (Oxford) Limited
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportSummit (Oxford) Limited
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSummit (Oxford) Limited
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address136a Eastern Avenue, Milton Park
    B.5.3.2Town/ cityAbingdon
    B.5.3.3Post codeOX14 4SB
    B.5.3.4CountryUnited Kingdom
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/08/591
    D.3 Description of the IMP
    D.3.2Product code SMT C1100
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPending
    D.3.9.1CAS number 945531-77-1
    D.3.9.2Current sponsor codeSMT C1100
    D.3.9.4EV Substance CodeSUB44065
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    An inherited disease causing muscle degeneration.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To investigate changes in leg MRI in paediatric patients with DMD, following treatment with SMT C1100(cohorts 1 and 2 )

    To investigate the relationships between changes in leg MRI with plasma concentrations of SMT C1100 and its metabolites in paediatric patients with DMD, following treatment with SMT C1100 (cohorts 1 and 2)

    To assess the safety and tolerability of SMT C1100 and its metabolites in paediatric patients with DMD
    E.2.2Secondary objectives of the trial
    To investigate changes in utrophin expression and muscle fibre regeneration in muscle, in paediatric patients with DMD, following treatment with SMT C1100 (cohorts 1 and 2 )
    To investigate the relationships between changes , utrophin expression and fibre regeneration in muscle and safety parameters with plasma concentrations of SMT C1100 and its metabolites in paediatric patients with DMD, following treatment with SMT C1100 (cohorts 1 and 2 )
    To investigate changes in pulmonary function tests in paediatric subjects with DMD, following treatment with SMT C1100
    To investigate the relationships between changes in pulmonary function tests with plasma concentrations of SMT C1100 and its metabolites in paediatric subjects with DMD, following treatment with SMT C1100
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients will be required to satisfy the following criteria at the screening visit:
    1. Be able to provide written informed consent/assent as per local requirements.
    2. Be male.
    3.Be aged ≥5 years <10 years of age (from 5th birthday to 10th birthday)-not applicable for cohort 3
    4. Have phenotypic evidence of dystrophinopathy based on the onset of characteristic clinical symptoms or signs (e.g., proximal muscle weakness, waddling gait, and Gowers' manoeuvre), an elevated serum creatinine kinase level, and ongoing difficulty with walking.
    5. Have prior confirmation of the DMD diagnosis through:Documentation of the presence of a mutation in the dystrophin gene as determined by gene sequencing from a laboratory certified by the College of American Pathologists, the Clinical Laboratory Improvement Act/Amendment or an equivalent organization. Or Documentation of the absence of dystrophin in the muscle (via biopsy).
    6. Be willing and able to comply with two muscle biopsy procedures (not applicable for cohort 3)
    7. Be able to undergo MRI examination.
    8. Patients must have used stable systemic corticosteroids (prednisone, prednisolone or deflazacort) for a minimum of 6 months immediately prior to the start of the Treatment Phase , with no significant change in dosage or dosing regimen (not related to body weight change) and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
    9. Have the ability to walk at least 300 meters unassisted during the screening 6MWD and be below the protocol-specified threshold for 80%-predicted 6MWD (not applicable for cohort 3)
    10. Have results of two 6MWD at Baseline determined as valid. The results of the second 6MWD must be within 20% of the first 6MWD (not applicable for cohort 3)
    11. Have cardiac ECHO measurements showing an ejection fraction of ≥ 55% and fractional shortening of ≥28% (not applicable for cohort 3)
    12. Confirmed screening laboratory values within the central laboratory ranges (haematology, renal and serum electrolyte parameters and serum chemistry parameters) or considered not clinically significant in the opinion of the Investigator. Variations in specific parameters expected in a DMD population (e.g., aspartate aminotransferase, alanine aminotransferase, alkaline phosphatase, lactate dehydrogenase and creatinine phosphokinase) classed by the Investigator as not clinically significant will not exclude the patient
    13. Be willing and able to comply with scheduled visits, drug administration plan, study procedures, laboratory tests and study restrictions.
    14.Have taken part in a prior SMT C1100 study (not applicable for cohorts 1 and 2 )
    E.4Principal exclusion criteria
    Patients will be excluded from the study if they satisfy the following
    criteria at the screening visit
    1. Have physical exam findings that in the investigator’s opinion should be exclusionary e.g., lower limb injury that may affect 6MWD performance.
    2. Have any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation or reinitiation) in prophylaxis/treatment for congestive heart failure (CHF) within 3 months prior to the start of study treatment.
    3. Have uncontrolled clinical symptoms and signs of CHF (American College of Cardiology/American Heart Association Stage C or Stage D).
    4.Have abnormal GLDH at baseline (>1.5 x ULN)
    5.Have abnormal coagulation times at baseline (>1.5 x ULN)
    6. Have an abnormal ECG e.g., a QTcF >500ms, left bundle-branch block or any other major conduction defect.
    7. Use beta blockers (however, if during the course of the study they are clinically indicated they can be initiated; not applicable for cohort 3 )
    8. Use herbal supplements and be unwilling to stop these for the duration of the study.
    9. Have a known hypersensitivity to any of the ingredients or excipients of the IMP. (microfluidised oral suspension F3, cohort 1 : Poloxamer 188, Methylparaben, Propylparaben, Hydroxypropylmethyl cellulose, Glycerol, Non crystallizing sorbitol [70%], Xanthan gum, Strawberry cream flavour [PHS-132963]);
    10.Have a known hypersensitivity to any of the ingredients or excipients of the IMP. Powder for oral suspension (F6): hypromellose acetate (not applicable to cohorts 1 and 3 )
    11. Have been exposed to another investigational drug or DMD interventional agent within 3 months prior to start of the Treatment Phase. Prior exposure to SMT C1100 or participation in an approved deflazacort access program (e.g FOR-DMD or ACCESS DMD clinical trials ) within this period would not exclude the patient (provided they have been on stable treatment for 6 months )
    12. Have a history of major surgical procedure within 12 weeks prior to the start of the Treatment Phase (week 1)
    13. Be undertaking ongoing immunosuppressive therapy (other than corticosteroids).
    14. Have an expectation of a major surgical procedure (e.g., scoliosis surgery) during the 12-month Treatment Phase of the study.
    15. Require daytime ventilator assistance.
    16. Have a prior or ongoing medical condition (e.g., concomitant illness, psychiatric condition, behavioural disorder, alcoholism, drug abuse), medical history, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
    17. Be dairy or lactose intolerant or have any other dietary restrictions that might interfere with the conduct of the study.
    18. Be a smoker, use other tobacco or nicotine products or be exposed to daily passive smoking (including parent/legal guardian, siblings) so as to minimize environmental factors causing CYP1A induction.
    19.Be using an approved DMD medication or anticipates using one during the duration of the study e.g., ataluren, idebenone, drisapersen, Exondys51. Patients who are taking part in the FOR-DMD and ACCESS DMD studies may be allowed to take part.
    20 Be using an inducer of CYP1A1, CYP1A2 .
    21. Be using a substrate of CYP2B6.
    22. All prescription, OTC, and herbal products that are known CYP2B6 sensitive substrates (e.g., buproprion, efavirenz) will be excluded 14 days prior to study conduct (beginning at sceening) through 14 days after study conduct completion. Please note, this is not an exhaustive list of CYP2B6 substrates and a discussion with the Medical Monitor may be warranted
    23.Be using drugs that have serotonergic, norepinephrinergic or dopaminergic activity (e.g., selective serotonin reuptake inhibitors, serotonin norepinephrine reuptake inhibitors, tricyclic antidepressants, tryptophan, dextromethorphan, meperidine, bupropion, nortriptyline, desipramine, doxepin, amoxapine, rasagiline, selegiline), or treatments used in attention deficit hyperactivity disorder such as Methylphenidate, and phenethylamine (PEA).
    24.Use of substrates of breast cancer resistance protein (e.g., omeprazole, rabeprazole, fluvastatin, methotrexate, mitoxantrone, imatinib, irinotecan, lapatinib, rosuvastatin, sulfasalazine, topotecan).
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to Weeks 12, 24, 36 and 48 in MRI leg muscles parameters(Cohorts 1 and 2)

    SMT C1100 and metabolite plasma concentrations at Weeks 1 (Days 1 and 7) as applicable (Cohorts 2 and 3 only) 4, 8 (Cohorts 1 and 2 only), 12, 24, 36 and 48

    • Safety data including:
    Treatment emergent AEs
    E.5.1.1Timepoint(s) of evaluation of this end point
    From baseline to Weeks 12, 24, 36 and 48
    E.5.2Secondary end point(s)
    Change from baseline to Week 24 or 48 in utrophin expression via muscle biopsy analysis (Cohorts 1 and 2)
    • Change from baseline to Week 24 or 48 in muscle regeneration biomarkers, via muscle biopsy analysis(Cohorts 1 and 2)
    • Change from baseline to Weeks 12, 24, 36 and 48 (Cohorts 1 and 2) and from baseline to Weeks 1, 24 and 48 (Cohort 3) in pulmonary function tests
    • Safety data including:
    • Vital signs (systolic and diastolic blood pressure and heart rate)
    • Physical examination
    • Twelve-lead electrocardiogram (ECG)
    • Echocardiogram (ECHO)
    • Pulmonary function tests (forced expiratory volume in 1 second [FEV1], forced vital capacity [FVC] maximum inspiratory pressure [MIP], maximum expiratory pressure [MEP] and peak expiratory flow [PEF]);Peak cough flow (PCF) (Cohort 3)Sniff nasal inspiratory pressure (SNIP) (Cohort 3

    • Safety laboratory evaluations (clinical chemistry, haematology (all 3 cohorts) coagulation (Cohort 2 and Cohort 3) parameters and urinalysis (all 3 cohorts)
    E.5.2.1Timepoint(s) of evaluation of this end point
    From baseline to Week 24 or 48
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Exploratory biomarkers
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned7
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months5
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 40
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F. of subjects for this age range: 40
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F. of subjects incapable of giving consent
    Subjects aged 5-10, parents/ legal guardians will be required to give
    Informed Consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state22
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 22
    F.4.2.2In the whole clinical trial 40
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    No IMP will be provided after the end of the Treatment Phase. However, subjects enrolled in this study may be eligible for studies investigating SMT C1100 in the future
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-01-19
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-09-11
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